BOSTON and LONDON - Centessa Pharmaceuticals plc (NASDAQ:CNTA), a clinical-stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application. This clearance paves the way for Phase 1 clinical trials of ORX750, a new drug candidate aimed at treating narcolepsy and potentially other sleep-wake disorders.
ORX750 is an orally administered, selective orexin receptor 2 (OX2R) agonist that targets the loss of orexin neurons, a condition associated with narcolepsy type 1 (NT1). The drug also shows promise for treating narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and similar disorders. The upcoming Phase 1 study will assess the drug's safety, tolerability, and pharmacokinetics in healthy adult volunteers, with dosing expected to commence shortly.
The trial will feature single-ascending doses (SAD) and multiple-ascending doses (MAD), as well as a cross-over pharmacodynamic (PD) assessment. This will include the Maintenance of Wakefulness Test (MWT) and the Karolinska Sleepiness Scale (KSS) to evaluate the drug's effects on sleep-deprived subjects. These tests are designed to provide early proof-of-concept data and assist in dose selection for subsequent trials in NT1, NT2, and IH patient populations.
The FDA has specified a maximum exposure limit for the study, which Centessa believes significantly exceeds the predicted efficacious doses of ORX750 for the targeted indications, indicating that the limit should not impact the planned clinical development activities for the drug.
Saurabh Saha, MD, PhD, CEO of Centessa, expressed optimism about the upcoming study, which he described as an "elegant, adaptive Phase 1 study" that is expected to yield proof-of-concept data in the latter half of 2024.
Centessa's approach to drug development focuses on creating transformative medicines for patients, with a portfolio that includes programs in hemophilia, immuno-oncology, and sleep-wake disorders. While the company has high hopes for ORX750, it is important to note that the drug's efficacy and safety have yet to be established and will depend on the outcomes of ongoing and future clinical trials.
The announcement of the FDA's IND clearance for ORX750 is based on a press release statement from Centessa Pharmaceuticals.
InvestingPro Insights
As Centessa Pharmaceuticals (NASDAQ:CNTA) garners attention with the FDA's approval of its IND application for ORX750, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, Centessa holds a market capitalization of $1.06 billion, a testament to the biotech firm's growth potential in the eyes of investors. Notably, the company's revenue over the last twelve months as of Q4 2023 stood at $6.85 million, with a remarkable gross profit margin of 100%. This indicates that Centessa is efficiently managing its cost of goods sold, although it's important to recognize that the company reported an operating loss of $171.28 million over the same period, reflecting the high costs associated with research and development in the biopharmaceutical industry.
Investors are also encouraged by the stock's strong return over the last year, with a 112.68% increase, signaling robust market confidence. However, an InvestingPro Tip highlights that analysts do not anticipate the company will be profitable this year, which is a critical consideration for those assessing the long-term viability of their investment. Furthermore, Centessa's liquid assets exceed short-term obligations, providing some financial cushioning as the company navigates the costly process of drug development and clinical trials.
For investors looking for more in-depth analysis and additional InvestingPro Tips, there are 10 more tips available on Centessa Pharmaceuticals at https://www.investing.com/pro/CNTA. These can provide further insights into the company's financial position and market expectations. To access these valuable resources, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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